Access to innovation: Has HTA hijacked the debate?

Society’s views should be taken into account at the earliest opportunity so that price is not the only consideration

How innovation is valued is a debate that is becoming largely technical and it is a consideration that is addressed almost exclusively by health economists from government agencies, or industry representatives with a health economics background. It is a process that has been arrived at largely through necessity as governments have wrestled with the challenge of ever-increasing healthcare expenditure.

Stakeholder involvementIn the current environment, there is a real risk that the views of important stakeholders will be overlooked when decisions are made about healthcare expenditure priorities, arguably the most important of which is society. So before delving deeper into cost-effectiveness discussions and the merits of different methodologies, there must be far greater public and patient involvement in the decision-making process.

No-one disputes the need for tough decision-making when it comes to prioritising healthcare resources and, indeed, in the allocation of resources to the provision of healthcare. Investment in healthcare is significant. The average expenditure across member states is already approaching 10 per cent of GDP and this figure is set to increase. A recent report from the European Commission's Directorate-General for Financial and Economic Affairs predicts that 'the pure demographic effect of an ageing population is projected to push up healthcare spending by between 1 and 2 per cent of GDP in most member states, i.e. an increase by approximately 25 per cent of current spending level'. Compare this to the 4 per cent average expenditure across member states invested in education and it is clear why the subject of affordability of healthcare is such a hot and current topic.

Gordon Spencer of Shire Pharmaceuticals states: “Affordability, particularly in a time of fiscal austerity, has become the watchword for health agencies around Europe and, to some extent, provides a mandate for limiting expenditure on new technology.

“However, there are inherent risks in abdicating responsibility for these decisions to advisory bodies alone and it is time that governments provided more thoughtful input into what society will pay for, based on much broader and earlier stakeholder engagement. In order to make headroom for those interventions likely to escape the narrow confines of cost-effectiveness measures, advisory bodies would benefit from clearer guidance on what society wants from a healthcare system.”

AffordabilityThe extent to which something is affordable is measured by its cost, relative to the amount that the purchaser is able to pay; what is affordable for one country may not be affordable for another. However, the quality of healthcare within a country cannot always be attributed to the level of healthcare expenditure alone.

That the citizens of Sweden can expect to live eight years longer than those of Latvia is more likely to be explained by basic health determinants such as health education and environmental factors, rather than just access to new technology, or the overall level of healthcare expenditure. In countries where healthcare is poor, investment priorities need to be channelled into more fundamental rights of citizens. In Western Europe, where life expectancy is high and has been growing for decades, patients' needs inevitably focus on the quality of life needed to match that longevity.

However, in all countries, those with rare, or some end-of-life, diseases are considered very costly, given the very modest quality of life and morbidity and mortality benefits that new interventions are likely to provide.

Acceptance levelsWith innovative treatments for these types of conditions, it will always be difficult to demonstrate acceptable incremental cost-effective ratios (ICER), for example. Yet, there is a genuine willingness across society to fund these innovations if an 'acceptable' level of effectiveness and safety has been demonstrated. A child with a rare genetic illness, if given access to a new treatment, will more often than not cost the state a disproportionate amount compared to the treatment of disease in the general population. Society accepts this and the expectation remains that treatment decisions should be made at an individual, as well as a population, level.

It is often argued that investment in interventions that are not 'cost-effective' must, somewhere else in the system, result in disinvestment to the detriment of others. This is oversimplistic in the extreme and overlooks the fact that healthcare systems have in-built inefficiencies and are generally wasteful. To use this argument for limiting expenditure is like building a skyscraper without lifts in order to come in on budget.

Government roleGovernments have a critical role in enabling those they represent to have a voice in what should, and what should not, be considered of value to society.

Michael Sobanja, chief officer at NHS Alliance says: “What we are prepared to pay for healthcare innovation needs to be better articulated by elected governments and set out as a framework against which technology assessments are based. These frameworks should be guided by thinking and policies that combine social and economic priorities. That virtually each member state uses different methodologies, leading to different recommendations, is perhaps testament to the difficulty in isolating the impact of any one intervention within a complex system. However, this only reinforces the need for effective public engagement and input.”

While there are country-specific advisory bodies that actively seek the public's input, such activity is by no means universal. Indeed, many governments abdicate their responsibilities while doing little to encourage advisory bodies to seek broader public opinion.

A public engagement programme that is arguably leading the way in Europe is the UK's National Institute for Health and Clinical Excellence (NICE) Citizens Council initiative. This is an excellent example of the right intention, although it has been perceived as a 'tick box' exercise by some. Notwithstanding this view, it is worthy of further consideration.

Established in 2002, the council comprises 30 ordinary members of the public. It provides NICE with advice that reflects the public's perspective, while ensuring the views of the public underpin the thoughts and processes of NICE. The council also evaluates the social and moral issues raised by NICE guidelines.

Although far from perfect, the principles behind this approach are ones that could, and indeed should, be replicated by governments and policy makers across Europe.

Supporting patient engagementVery few Health Technology Assessment (HTA) agencies or advisory bodies involve patients or consider the patient perspective as part of their outputs. There is a multitude of reasons for this, probably including tokenism, a lack of effort on the part of regulators, a lack of effective advocacy networks and, for the patient group, the sheer complexity of the area.But arguably, the challenge in eliciting appropriate patient input runs far deeper than this. The problem is that the debate begins at the technical level. Advisory bodies such as HTA agencies, and those companies who interact with them, begin with an intervention and then try to assess its future value. The techniques involved are complex, and the interpretation of statistical modelling forms the basis of discussion, rather than an assessment of the needs gathered from broad stakeholder engagement prior to technical assessments.

Alex Wyke, CEO of PatientView, an independent organisation that works closely with patients and with health and social campaigning groups worldwide, states: “None of us, outside of a small group of health economists, is able to effectively engage in discussions on assessing the value of innovation – it's just too complex. Indeed, governments themselves are just as incapable of challenging the process. It is time that 'what we value' becomes the starting point for discussion and agreement, and then HTAs can be carried out according to these overarching goals.”

Industry roleIndustry must shoulder its proportion of the overall responsibility to drive change too. Pricing of products needs to reflect value and while publicly executives will say this is what happens, privately many will acknowledge that pricing decisions start with research based upon what the market will bear. Pricing is one of the 'four Ps' of marketing, yet it is rarely used by industry to increase volume share; too often a narrow positioning for a premium price is accepted. This in turn can limit patients' access to the medicines they need.

So what practical steps can the industry take to help ensure the HTA process considers wider societal views on the value of innovation and that the voice of the patient is heard? It could start by leading by example.

Earlier engagement with patient representatives during the early development phases of a drug will offer unique insights into the value patients may put on a particular intervention. This will then help guide the clinical development programme. Ongoing dialogue through the late development phase will provide additional understanding of patients' attitudes and behaviours, information that is critical in bringing an asset to market backed by programmes that support patient concordance and persistence.

Patient's voiceThe industry is also highly proficient at facilitating interactions between disparate stakeholders at all levels of healthcare systems. These skills could be deployed to good effect in bringing patients into the HTA arena.

So has HTA hijacked the debate? The answer is no. Hijacking is, by its very nature, the seizing of something by a person or organisation. The current situation has evolved and, although far from ideal, steps can still be taken to redress the balance to ensure that society's voice is heard.

It is widely accepted that health is important for the wellbeing of individuals and society, and that a healthy population is also a prerequisite for economic productivity and prosperity. Indeed, as recognised by the European Commission: 'health is the greatest wealth'.